Autism is a cognitive disorder associated with three diagnostic characteristics: abnormal social interaction, impaired communication and repetitive patterns of behavior (Geshwind D H and Levitt P, Curr. Opin. Neurobiol. 17:103, 2007). Autism is part of a spectrum of disorders called autism spectrum disorders (ASD). Autism has an incidence of approximately 1 in 1,000 individuals while ASD has a higher incidence of 1 to 6 per 1000 individuals. Onset is typically prior to three years of age.
There is a consensus that autism is a neurobiological disorder (Luke Y and Tsai M D, Psychosomatic Medicine 61:651, 1999; Levy & Schultz, Lancet 374:1627, 2009). However, no specific neurological or biological marker has been uncovered as the cause of autism and the etiology of autism remains unknown. To date, there is no etiology-based treatment for autism or ASD. The recommended medical intervention is behavioral rather than pharmacological. Physicians and parents of patients with autism focus on approaches such as behavioral modification, training in social skills and speech therapy.
A number of psychopharmacological agents are utilized in children with autism, but only to address specific psychiatric or behavioral symptoms. These agents include: serotonine-related drugs (buspirone, clomipramine, fenfluramine, fluoxetine, fluvoxamine, sertraline), dopamine-related agents (haloperidol, L-dopa, pimozide), epinephrine and norepinephrine-related compounds (beta-blockers, clonidine, desipramine), and a variety of other agents such as opiate antagonists, ACTH, clozapine, risperidone, vitamins B6 and B12 and melatonin.
Thus, while children with ASD are prescribed a number of drugs, there is still no accepted rational therapeutic paradigm designed to address autism or its causes.
Stiripentol (Diacomit, 1-penten-3-ol, 1-(1,3-benzodioxol)-4,4-dimethyl or 4-dimethyl-1-[3,4-methylenedioxy-3,4)-phenyl]-1-penten-3-ol) is a racemic allylic alcohol that is structurally unrelated to other antiepileptic drugs.

Stiripentol has shown anticonvulsant activity in several animal models but its spectrum of clinical activity is relatively narrow. Stiripentol has exhibited a high response rate in SMEI patients at a dose of 50 mg/kg/day. Recently, stiripentol has shown high efficacy in two double blind controlled clinical trials and has received approval from the European Medicines Agency (Chiron C. Lancet 356:1638, 2000).